2010. FDA still has many complex questions to consider in implementing BPCIA. Even
after it issues regulations, it will take time for the agency to prepare specific written
requests, for willing sponsors to conduct and submit requested studies, and then for FDA
to evaluate the submissions and make its judgments public. Given these constraints, the
committee concluded that it was too early either to assess the impact of BPCIA on
pediatric studies of biologics or to reach conclusions about its effectiveness or its
limitations in ensuring pediatric studies of biologics.
Thus, it is reasonable for Congress
to continue the extension of BPCA to biologics until the results can be systematically
evaluated 3 to 5 years after FDA issues implementing regulations.
Barring surprises in their implementation, the incentives of BPCIA can be
expected to encourage further pediatric studies of both older and newer biologics.
Nonetheless, it seems unlikely that the law will lead to a surge of written requests for
pediatric studies of biologics similar to the surge in requests for pediatric drug studies
that followed the creation of the pediatric exclusivity incentive in 1997. Since 1999,
biologics have been subject to PREA requirements (with exemptions for orphandesignated drugs). In addition, biologics have been eligible for the incentives of the
Orphan Drug Act, which offer 7 years of exclusivity. Nearly three-quarters of the 390-
plus orphan drug and biologics approvals since 1984 have involved rare conditions that
affect children.
Whether as a result of PREA, the Orphan Drug Act, the evident therapeutic
promise of many biologics, or other factors, approximately 60 percent of the 97 stillmarketed biologics (excluding vaccines, assays, and reagents) that FDA has approved
since 1997 are labeled for pediatric use, have some information about pediatric studies in
the labeling, or have warnings against pediatric use based on analysis of postmarket
safety reports. Further, an examination of studies registered at the ClinicalTrials.gov
database indicates that most of the remaining products have been studied, are being
studied, or are planned for studies with children. Of the dozen biologics that have not
been studied with children, most appear either to have limited potential to benefit
children or to be in the same class as alternative products that are labeled for pediatric
use. On the basis of case reports of off-label use and other information, the committee
identified one product that may have sufficient promise for treating refractory infantile
hemangiomas that FDA or NIH, or both, might consider encouraging or supporting
controlled pediatric trials of its safety and efficacy.
The committee’s finding that most biologics have been studied with children does
not mean that no further opportunities or needs for pediatric studies of these medications
exist. Such opportunities could include studies that pursue promising findings in early